The drug, Givinostat, has been approved as safe for use but Belfast Health Trust, NI's specialist centre for the condition, ...

Muscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, causing ...

Sarepta Therapeutics ( NASDAQ: SRPT) reported a second fatality from acute liver failure in a patient treated with its gene ...

Sarepta suspends Elevidys use in non-ambulatory Duchenne patients after two liver failure deaths, triggering FDA review and ...

Liver damage is a well-recognised complication of gene therapies based on adeno-associated virus (AAV) vectors, and other ...

Roche (SIX:ROG) shares dropped 2% on Monday after the company paused dosing of its gene therapy Elevidys in non-ambulatory ...

The commercial and clinical use of Elevidys was discontinued after two fatal cases of acute liver failure.

U.S. FDA successfully completed Pre-License Inspection; Company expects facility will meet all requirements to support ...

Shares of Sarepta Therapeutics plunged 34% in premarket trading on Monday, a day after the company disclosed a second case of patient death due to acute liver failure after receiving its gene therapy ...

Sarepta said it was halting shipments of its Duchenne gene therapy for patients who can no longer walk, following the death ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety ...

Sarepta Therapeutics shares dived 42% in premarket trading after the company disclosed a second reported case of acute liver failure that resulted in a death tied to its Duchenne muscular dystrophy ...